Advanced Therapies
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News
NEW REVIEW | Treatment Landscape of Rare Anaemias Is Shifting Rapidly
Writing in Frontiers in Medicine, the authors began by reviewing congenital and acquired forms of anaemia. Those include sickle cell disease (SCD) and hemolytic anemias (CHAs), both congenital, and autoimmune hemolytic…
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TOP STORY | EMA Validates Vertex’s/CRISPR Submission Of Exa-Cel For β-Τhal And SCD
The Marketing Authorization Application (MAA) for exagamglogene autotemcel (exa-cel), marks the first regulatory submission in the EU for a CRISPR-based medicine. Through the validation, exa-cel is indicated for the…
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NEW | Bluebirdbio Releases Resources For Patients On Gene Therapy Zynteglo®
To help people who are living with or caring for someone who has β-thalassaemia major get substantial information about Zynteglo®, bluebird bio, the company behind this ground-breaking therapy, has recently…
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ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.…
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POLICY NEWS | TIF Issues Statement On The New EU Regulation For Blood, Tissues And Cells
This single Regulation, once approved by Member States, will replace all existing EU Directives on Blood, Tissues and Cells and will be equally applicable in the EU territory. The proposal…
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